反应停最初是用于治疗妊娠妇女呕吐的镇静药，但在20世纪60年代因发现引起胎儿肢体畸形而被禁用，退出市场。如今，研究人员发现，这种药可用于治疗一种影响血管的遗传性疾病。
遗传性出血性毛细血管扩张症（HHT）是一种源自遗传的血管畸形症，许多患者会出现重复性且难治疗的鼻出血，严重者会影响到生活质量。Franck Lebrin和同事发现，用反应停治疗这种患者会降低其鼻出血的严重程度和发生频率。在对HHT模型小鼠的实验中，通过一种包含生长因子PDGF的机制，反应停治疗救治了有缺陷血管壁的模型小鼠。取自HHT患者血管壁的生物切片显示，类似的机制也许也能解释反应停对人类的治疗效果。
科学家们发现，反应停除了对HHT患者具有治疗效果外，还可用于某类癌症的治疗，表明这种隐退已久的著名药物出现了新的生机。 该成果发表在《自然―医学》（Natural Medicine）上。
推荐原文出处：
Nature Medicine doi:10.1038/nm.2131
Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia
Franck Lebrin,Samly Srun,Karine Raymond,Sabrina Martin,Stieneke van den Brink,Catarina Freitas,Christiane Bréant,Thomas Mathivet,Bruno Larrivée,Jean-Léon Thomas,Helen M Arthur,Cornelis J J Westermann,Frans Disch,Johannes J Mager,Repke J Snijder,Anne Eichmann" Christine L Mummery
Hereditary hemorrhagic telangiectasia (HHT) is an inherited disorder characterized by vascular malformations. Many affected individuals develop recurrent nosebleeds, which can severely affect their quality of life and are clinically difficult to treat. We report here that treatment with thalidomide reduced the severity and frequency of nosebleeds (epistaxis) in the majority of a small group of subjects with HHT tested. The blood hemoglobin levels of the treated individuals rose as a result of reduced hemorrhage and enhanced blood vessel stabilization. In mice heterozygous for a null mutation in the Eng gene (encoding endoglin), an experimental model of HHT, thalidomide treatment stimulated mural cell coverage and thus rescued vessel wall defects. Thalidomide treatment increased platelet-derived growth factor-B (PDGF-B) expression in endothelial cells and stimulated mural cell activation. The effects of thalidomide treatment were partially reversed by pharmacological or genetic interference with PDGF signaling from endothelial cells to pericytes. Biopsies of nasal epithelium from individuals with HHT treated or not with thalidomide showed that similar mechanisms may explain the effects of thalidomide treatment in humans. Our findings demonstrate the ability of thalidomide to induce vessel maturation, which may be useful as a therapeutic strategy for the treatment of vascular malformations.
